In drug development activities, efficacy studies are typically performed before a candidate drug is evaluated for toxicity. A drug may combine with a known receptor and demonstrate some biochemical reaction at the cell level. Efficacy studies are generally performed in advance of clinical evaluation on humans where the drug may be deemed unsatisfactory due to toxic effects. Thus substantial investments of cost and time occurring prior to human clinical trials may be at risk.
Organoids are three-dimensional miniaturized versions of an organ that can be produced in vitro. Organoids may be produced from one or more stem cells that organize into larger cell organizations based on the self-renewal and differentiation capabilities of the stem cells. If the organoids are sufficiently similar to a corresponding human organ in terms of susceptibility to a drug candidate, it may be possible to evaluate the response of similar organoids in parallel with the main efficacy process. For example, drug discovery processes for neurological diseases or states can be correlated with the degree of neurotransmitter synthesis. The process may look for candidates that combine with a receptor to change or modulate the generation of these neurotransmitters based on knowledge about the disease. The ability to generate appropriate clumps of cells for this purpose is difficult. Researchers want to generate homogeneous populations of organoids that correspond to a specific portion of the brain; however, efforts generally only result in the generation of heterogeneous populations representing multiple regions of the brain.